JP2014515762A5 - - Google Patents
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- JP2014515762A5 JP2014515762A5 JP2014509281A JP2014509281A JP2014515762A5 JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5 JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014509281 A JP2014509281 A JP 2014509281A JP 2014515762 A5 JP2014515762 A5 JP 2014515762A5
- Authority
- JP
- Japan
- Prior art keywords
- conjugate
- occurrence
- independently
- amino acid
- carrier peptide
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
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- 239000000562 conjugate Substances 0.000 claims description 118
- 235000001014 amino acid Nutrition 0.000 claims description 74
- 229940024606 amino acid Drugs 0.000 claims description 74
- 150000001413 amino acids Chemical class 0.000 claims description 70
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 53
- 125000000217 alkyl group Chemical group 0.000 claims description 34
- 229910052739 hydrogen Inorganic materials 0.000 claims description 31
- 239000001257 hydrogen Substances 0.000 claims description 30
- 102000039446 nucleic acids Human genes 0.000 claims description 23
- 108020004707 nucleic acids Proteins 0.000 claims description 23
- 150000007523 nucleic acids Chemical class 0.000 claims description 23
- 239000004475 Arginine Substances 0.000 claims description 18
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims description 18
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical compound NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 claims description 16
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 14
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims description 14
- 150000002431 hydrogen Chemical class 0.000 claims description 14
- 239000004471 Glycine Substances 0.000 claims description 12
- 230000002209 hydrophobic effect Effects 0.000 claims description 12
- 238000000034 method Methods 0.000 claims description 12
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims description 12
- 125000004448 alkyl carbonyl group Chemical group 0.000 claims description 11
- ONIBWKKTOPOVIA-UHFFFAOYSA-N Proline Natural products OC(=O)C1CCCN1 ONIBWKKTOPOVIA-UHFFFAOYSA-N 0.000 claims description 10
- 125000003118 aryl group Chemical group 0.000 claims description 10
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 10
- ONIBWKKTOPOVIA-BYPYZUCNSA-N L-Proline Chemical compound OC(=O)[C@@H]1CCCN1 ONIBWKKTOPOVIA-BYPYZUCNSA-N 0.000 claims description 8
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims description 8
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims description 8
- 239000004472 Lysine Substances 0.000 claims description 8
- 210000004899 c-terminal region Anatomy 0.000 claims description 8
- -1 glycine amino acid Chemical class 0.000 claims description 8
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims description 8
- IJGRMHOSHXDMSA-UHFFFAOYSA-N Atomic nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims description 6
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 claims description 6
- 125000003342 alkenyl group Chemical group 0.000 claims description 6
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 6
- 125000003630 glycyl group Chemical group [H]N([H])C([H])([H])C(*)=O 0.000 claims description 6
- 125000000623 heterocyclic group Chemical group 0.000 claims description 6
- 229910052760 oxygen Inorganic materials 0.000 claims description 6
- 125000006552 (C3-C8) cycloalkyl group Chemical group 0.000 claims description 5
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- 244000007835 Cyamopsis tetragonoloba Species 0.000 claims description 4
- RTZKZFJDLAIYFH-UHFFFAOYSA-N Diethyl ether Chemical compound CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims description 4
- COLNVLDHVKWLRT-QMMMGPOBSA-N L-phenylalanine Chemical compound OC(=O)[C@@H](N)CC1=CC=CC=C1 COLNVLDHVKWLRT-QMMMGPOBSA-N 0.000 claims description 4
- GLUUGHFHXGJENI-UHFFFAOYSA-N Piperazine Chemical compound C1CNCCN1 GLUUGHFHXGJENI-UHFFFAOYSA-N 0.000 claims description 4
- 208000036142 Viral infection Diseases 0.000 claims description 4
- 125000003545 alkoxy group Chemical group 0.000 claims description 4
- 125000003282 alkyl amino group Chemical group 0.000 claims description 4
- 125000000304 alkynyl group Chemical group 0.000 claims description 4
- 125000004103 aminoalkyl group Chemical group 0.000 claims description 4
- 150000001484 arginines Chemical class 0.000 claims description 4
- 125000000637 arginyl group Chemical group N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims description 4
- 125000004429 atom Chemical group 0.000 claims description 4
- UCMIRNVEIXFBKS-UHFFFAOYSA-N beta-alanine Chemical group NCCC(O)=O UCMIRNVEIXFBKS-UHFFFAOYSA-N 0.000 claims description 4
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 4
- 210000004027 cell Anatomy 0.000 claims description 4
- ANCLJVISBRWUTR-UHFFFAOYSA-N diaminophosphinic acid Chemical compound NP(N)(O)=O ANCLJVISBRWUTR-UHFFFAOYSA-N 0.000 claims description 4
- 125000001072 heteroaryl group Chemical group 0.000 claims description 4
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 4
- 125000005647 linker group Chemical group 0.000 claims description 4
- 208000018360 neuromuscular disease Diseases 0.000 claims description 4
- 229910052757 nitrogen Inorganic materials 0.000 claims description 4
- 239000000863 peptide conjugate Substances 0.000 claims description 4
- COLNVLDHVKWLRT-UHFFFAOYSA-N phenylalanine Natural products OC(=O)C(N)CC1=CC=CC=C1 COLNVLDHVKWLRT-UHFFFAOYSA-N 0.000 claims description 4
- 150000003839 salts Chemical class 0.000 claims description 4
- 229910052717 sulfur Inorganic materials 0.000 claims description 4
- 230000009385 viral infection Effects 0.000 claims description 4
- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims description 2
- 125000004642 (C1-C12) alkoxy group Chemical group 0.000 claims description 2
- SLXKOJJOQWFEFD-UHFFFAOYSA-N 6-aminohexanoic acid Chemical group NCCCCCC(O)=O SLXKOJJOQWFEFD-UHFFFAOYSA-N 0.000 claims description 2
- DCXYFEDJOCDNAF-UHFFFAOYSA-N Asparagine Natural products OC(=O)C(N)CC(N)=O DCXYFEDJOCDNAF-UHFFFAOYSA-N 0.000 claims description 2
- 208000035143 Bacterial infection Diseases 0.000 claims description 2
- KXDHJXZQYSOELW-UHFFFAOYSA-M Carbamate Chemical compound NC([O-])=O KXDHJXZQYSOELW-UHFFFAOYSA-M 0.000 claims description 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 2
- 206010061218 Inflammation Diseases 0.000 claims description 2
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical compound SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 claims description 2
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 claims description 2
- DCXYFEDJOCDNAF-REOHCLBHSA-N L-asparagine Chemical compound OC(=O)[C@@H](N)CC(N)=O DCXYFEDJOCDNAF-REOHCLBHSA-N 0.000 claims description 2
- ZDXPYRJPNDTMRX-VKHMYHEASA-N L-glutamine Chemical compound OC(=O)[C@@H](N)CCC(N)=O ZDXPYRJPNDTMRX-VKHMYHEASA-N 0.000 claims description 2
- FFEARJCKVFRZRR-BYPYZUCNSA-N L-methionine Chemical compound CSCC[C@H](N)C(O)=O FFEARJCKVFRZRR-BYPYZUCNSA-N 0.000 claims description 2
- AYFVYJQAPQTCCC-GBXIJSLDSA-N L-threonine Chemical compound C[C@@H](O)[C@H](N)C(O)=O AYFVYJQAPQTCCC-GBXIJSLDSA-N 0.000 claims description 2
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 2
- AYFVYJQAPQTCCC-UHFFFAOYSA-N Threonine Natural products CC(O)C(N)C(O)=O AYFVYJQAPQTCCC-UHFFFAOYSA-N 0.000 claims description 2
- 239000004473 Threonine Substances 0.000 claims description 2
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 claims description 2
- 235000004279 alanine Nutrition 0.000 claims description 2
- 125000004453 alkoxycarbonyl group Chemical group 0.000 claims description 2
- 235000008206 alpha-amino acids Nutrition 0.000 claims description 2
- 150000001408 amides Chemical class 0.000 claims description 2
- 125000000747 amidyl group Chemical group [H][N-]* 0.000 claims description 2
- 229960002684 aminocaproic acid Drugs 0.000 claims description 2
- 125000005098 aryl alkoxy carbonyl group Chemical group 0.000 claims description 2
- 125000005099 aryl alkyl carbonyl group Chemical group 0.000 claims description 2
- 125000005129 aryl carbonyl group Chemical group 0.000 claims description 2
- 125000005161 aryl oxy carbonyl group Chemical group 0.000 claims description 2
- 125000004104 aryloxy group Chemical group 0.000 claims description 2
- 235000009582 asparagine Nutrition 0.000 claims description 2
- 229960001230 asparagine Drugs 0.000 claims description 2
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical compound [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 2
- 208000022362 bacterial infectious disease Diseases 0.000 claims description 2
- 125000003236 benzoyl group Chemical group [H]C1=C([H])C([H])=C(C([H])=C1[H])C(*)=O 0.000 claims description 2
- 229940000635 beta-alanine Drugs 0.000 claims description 2
- 125000002618 bicyclic heterocycle group Chemical group 0.000 claims description 2
- 230000027455 binding Effects 0.000 claims description 2
- 150000001721 carbon Chemical class 0.000 claims description 2
- 125000004432 carbon atom Chemical group C* 0.000 claims description 2
- 230000001268 conjugating effect Effects 0.000 claims description 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 2
- 125000005170 cycloalkyloxycarbonyl group Chemical group 0.000 claims description 2
- 235000018417 cysteine Nutrition 0.000 claims description 2
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims description 2
- 229940009976 deoxycholate Drugs 0.000 claims description 2
- KXGVEGMKQFWNSR-LLQZFEROSA-N deoxycholic acid Chemical compound C([C@H]1CC2)[C@H](O)CC[C@]1(C)[C@@H]1[C@@H]2[C@@H]2CC[C@H]([C@@H](CCC(O)=O)C)[C@@]2(C)[C@@H](O)C1 KXGVEGMKQFWNSR-LLQZFEROSA-N 0.000 claims description 2
- 150000002148 esters Chemical class 0.000 claims description 2
- ZDXPYRJPNDTMRX-UHFFFAOYSA-N glutamine Natural products OC(=O)C(N)CCC(N)=O ZDXPYRJPNDTMRX-UHFFFAOYSA-N 0.000 claims description 2
- 235000004554 glutamine Nutrition 0.000 claims description 2
- 125000005842 heteroatom Chemical group 0.000 claims description 2
- 125000004435 hydrogen atom Chemical group [H]* 0.000 claims description 2
- 230000004054 inflammatory process Effects 0.000 claims description 2
- 206010022000 influenza Diseases 0.000 claims description 2
- 229930182817 methionine Natural products 0.000 claims description 2
- 125000002950 monocyclic group Chemical group 0.000 claims description 2
- 230000007935 neutral effect Effects 0.000 claims description 2
- QJGQUHMNIGDVPM-UHFFFAOYSA-N nitrogen group Chemical group [N] QJGQUHMNIGDVPM-UHFFFAOYSA-N 0.000 claims description 2
- 125000004043 oxo group Chemical group O=* 0.000 claims description 2
- 239000001301 oxygen Substances 0.000 claims description 2
- 150000004713 phosphodiesters Chemical class 0.000 claims description 2
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims description 2
- 208000030761 polycystic kidney disease Diseases 0.000 claims description 2
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims description 2
- 230000009870 specific binding Effects 0.000 claims description 2
- 125000003696 stearoyl group Chemical group O=C([*])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims description 2
- 230000008685 targeting Effects 0.000 claims description 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 2
- 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 claims description 2
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 125000004183 alkoxy alkyl group Chemical group 0.000 description 1
- 230000021615 conjugation Effects 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/101,942 | 2011-05-05 | ||
| US13/101,942 US20110269665A1 (en) | 2009-06-26 | 2011-05-05 | Compound and method for treating myotonic dystrophy |
| US13/107,528 US9238042B2 (en) | 2010-05-13 | 2011-05-13 | Antisense modulation of interleukins 17 and 23 signaling |
| US13/107,528 | 2011-05-13 | ||
| PCT/US2011/061282 WO2012150960A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016151179A Division JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Division JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2014515762A JP2014515762A (ja) | 2014-07-03 |
| JP2014515762A5 true JP2014515762A5 (enExample) | 2015-01-29 |
| JP6478632B2 JP6478632B2 (ja) | 2019-03-06 |
Family
ID=45218873
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014509281A Active JP6478632B2 (ja) | 2011-05-05 | 2011-11-17 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2016151179A Pending JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Withdrawn JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2020070566A Active JP6884250B2 (ja) | 2011-05-05 | 2020-04-09 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2021080358A Pending JP2021113232A (ja) | 2011-05-05 | 2021-05-11 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2024014898A Pending JP2024032974A (ja) | 2011-05-05 | 2024-02-02 | ペプチドオリゴヌクレオチドコンジュゲート |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016151179A Pending JP2016185991A (ja) | 2011-05-05 | 2016-08-01 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2018108772A Withdrawn JP2018135396A (ja) | 2011-05-05 | 2018-06-06 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2020070566A Active JP6884250B2 (ja) | 2011-05-05 | 2020-04-09 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2021080358A Pending JP2021113232A (ja) | 2011-05-05 | 2021-05-11 | ペプチドオリゴヌクレオチドコンジュゲート |
| JP2024014898A Pending JP2024032974A (ja) | 2011-05-05 | 2024-02-02 | ペプチドオリゴヌクレオチドコンジュゲート |
Country Status (8)
| Country | Link |
|---|---|
| EP (1) | EP2704749A1 (enExample) |
| JP (6) | JP6478632B2 (enExample) |
| KR (3) | KR102339196B1 (enExample) |
| CN (2) | CN107693797B (enExample) |
| AU (6) | AU2011367230B2 (enExample) |
| CA (2) | CA2834128A1 (enExample) |
| IL (2) | IL273838B (enExample) |
| WO (1) | WO2012150960A1 (enExample) |
Families Citing this family (90)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3808845A1 (en) | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| EP2049664B1 (en) | 2006-08-11 | 2011-09-14 | Prosensa Technologies B.V. | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| CA3066050A1 (en) | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| SI2499249T1 (sl) | 2009-11-12 | 2019-02-28 | The University Of Western Australia, | Protismiselne molekule in postopki zdravljenja patologij |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| HK1201514A1 (en) | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| AU2012345638C1 (en) | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| ES2832531T3 (es) | 2011-11-30 | 2021-06-10 | Sarepta Therapeutics Inc | Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones |
| US9326992B2 (en) | 2011-12-08 | 2016-05-03 | Sarepta Therapeutics, Inc. | Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| PL2841578T3 (pl) | 2012-04-23 | 2017-12-29 | Biomarin Technologies B.V. | Oligonukleotydy modulujące RNA o ulepszonych właściwościach do leczenia zaburzeń nerwowo-mięśniowych |
| EP3885439A1 (en) | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| EP2946013A1 (en) | 2013-01-16 | 2015-11-25 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| EP3760720A1 (en) | 2013-03-14 | 2021-01-06 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| IL280443B (en) | 2013-03-14 | 2022-07-01 | Sarepta Therapeutics Inc | Preparations that skip axon for the treatment of muscular dystrophy |
| MX373959B (es) | 2013-03-15 | 2020-07-13 | Sarepta Therapeutics Inc | Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). |
| CN105228999B (zh) * | 2013-05-24 | 2021-03-02 | 味之素株式会社 | 吗啉代寡核苷酸的制备方法 |
| TWI736514B (zh) * | 2013-09-05 | 2021-08-21 | 美商薩羅塔治療公司 | 酸性α葡萄糖苷酶之反義股誘導之外顯子２包含 |
| WO2015097600A2 (en) | 2013-12-24 | 2015-07-02 | Sentiss Pharma Private Limited | Topical brimonidine tartrate ophthalmic solution |
| WO2015137409A1 (ja) | 2014-03-12 | 2015-09-17 | 日本新薬株式会社 | アンチセンス核酸 |
| KR101661277B1 (ko) * | 2014-03-17 | 2016-09-30 | 제주광어주식회사 | 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스 |
| EP3620178A3 (en) | 2014-05-16 | 2020-07-22 | Oregon State University | Antisense antibacterial compounds and methods |
| US10391098B2 (en) | 2014-05-19 | 2019-08-27 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| JP6994941B2 (ja) | 2014-12-31 | 2022-02-04 | オレゴン ステート ユニバーシティ | アンチセンス抗細菌性化合物および方法 |
| US20180216111A1 (en) * | 2015-02-27 | 2018-08-02 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2016187425A1 (en) * | 2015-05-19 | 2016-11-24 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| MA50829A (fr) | 2015-06-01 | 2018-04-11 | Sarepta Therapeutics Inc | Exclusion d'exon induite pat technologie antisens dans le collagène de type vii |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| KR20250005537A (ko) * | 2015-08-05 | 2025-01-09 | 에자이 알앤드디 매니지먼트 가부시키가이샤 | 균질한 올리고머의 제조를 위한 키랄 시약 |
| HK1257498A1 (zh) * | 2015-08-28 | 2019-10-25 | Sarepta Therapeutics, Inc. | 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体 |
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| MA45819A (fr) | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
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